Marisa Wexler, MS,  senior science writer—

The U.S. Food and Drug Administration (FDA) has placed a hold on the 4D-310 clinical program, an experimental gene therapy for Fabry disease being developed by 4D Molecular Therapeutics (4DMT). The hold was disclosed in a filing submitted by 4DMT to the Securities and Exchange Commission earlier this month.

One year after treatment with 4D-310, an experimental gene therapy for Fabry disease, measures of heart health have improved in the first three participants in a clinical trial, according to new data announced by the therapy’s developer, 4D Molecular Therapeutics (4DMT). “4D-310 holds promise as a potential first-in-class treatment…

A program that screened nearly 500 men with implanted pacemakers for Fabry disease found that none of them had the genetic disorder. The findings “suggest that the prevalence of [Fabry disease] in male patients requiring permanent pacing is very low and does not justify a systematic screening,” according to…

ISU303, an experimental enzyme replacement therapy for Fabry disease, showed a good safety profile and some promising signs of efficacy in a small clinical trial in Asia, according to researchers. The “results suggest that ISU303 is safe and effective and [may be an] alternative ERT for [Fabry disease],” the…

One-time treatment with ST-920 (isaralgagene civaparvovec), an experimental gene therapy being developed by Sangamo Therapeutics, continues to be generally well-tolerated among people with Fabry disease, according to new data from the Phase 1/2 STAAR clinical trial. Trial data show that the gene therapy leads to long-term increases in activity…

Implementing nationwide screening programs for Fabry disease in people with a heart condition called hypertrophic cardiomyopathy (HCM) may help improve diagnosis of the disease, a study in Czech Republic shows. In the country, the program led to the diagnosis of Fabry in four men and two women among the…

Activating a mitochondrial protein called TRAP1 can help to improve the functionality of lysosomes, the cellular structures that are defective in Fabry disease, a new study reports. The study, “Activation of mitochondrial TRAP1 stimulates mitochondria-lysosome crosstalk and correction of lysosomal dysfunction,” was published in iScience.

AL01211, a once-daily oral therapy being developed by AceLink Therapeutics for Fabry disease, has been granted an orphan drug designation by the U.S. Food and Drug Administration (FDA). The FDA gives this designation to investigational treatments with the potential to improve care for rare diseases that affect fewer than…

Doctors who treat Fabry disease tend to underappreciate the effect that symptoms like abnormal sweating and digestive complaints can have on a person’s quality of life, according to a new study conducted in Japan. The study also highlighted a disconnect in communication between doctors and patients when it comes…

Mendelics and Takeda are partnering for a new program that aims to use a genetic sequencing strategy to make diagnosis of Fabry disease easier and faster. Fabry disease is caused by mutations in the GLA gene located on the X chromosome. This gene provides instructions for making the alpha-galactosidase…